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Desmopressin for Bedwetting in Children With SCD

Primary Purpose

Nocturnal Enuresis, Anemia, Sickle Cell

Status
Terminated
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Desmopressin
Sponsored by
Montefiore Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Nocturnal Enuresis

Eligibility Criteria

8 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Patients with Hemoglobin SS, SC, SB0thal or SB+thal
  2. Patients with at least two episodes of primary nocturnal enuresis per week or four episodes over the two weeks prior to enrollment.
  3. Patients with secondary enuresis who have been evaluated and cleared by a pediatric urologist as not having other etiologies of enuresis (e.g. overactive detrusor activity, a genitourinary anatomic abnormality)

Exclusion Criteria:

  1. Patients with developmental delay or neurologic dysfunction secondary to stroke.
  2. Patients with hypertension or underlying renal disease.
  3. Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing normal genitourinary anatomy is sufficient to clear a patient for the study.
  4. Patients with daytime urinary incontinence
  5. Patients with glucosuria on urinalysis.
  6. Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric urologist to rule out other etiologies of enuresis.
  7. Patients who are pregnant.
  8. Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).

Sites / Locations

  • Children's Hospital at Montefiore

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment Group

Arm Description

Desmopressin 0.2mg tablets, dose titrated to effect

Outcomes

Primary Outcome Measures

Change in Bedwetting episodes
To prospectively assess if the use of desmopressin in patients with sickle cell disease and nocturnal enuresis will decrease the number of nighttime episodes of enuresis by 50% after initiating DDAVP at 0.4 mg nightly dose with dose escalation as clinically appropriate.

Secondary Outcome Measures

Impact of Bedwetting on day to day activities
To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to their baseline. This will be measured using the PedsQL Measurement Model which measures health related quality of life in children with acute and chronic health conditions, like sickle cell. The scales focuses on areas such as activities, feelings, and school performance.
Change in Nighttime awakenings
To determine if the use of desmopressin in patients with nocturnal enuresis improves rates of nocturia, defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to prior to initiating treatment with DDAVP. Patient's will track their nighttime awakenings using a study diary.
Change in Daytime Fatigue
To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to their baseline data. The PROMIS Pediatric Fatigue Short Form will be used to compare levels of fatigue from baseline to 4 weeks on the medication.

Full Information

First Posted
May 21, 2020
Last Updated
June 12, 2023
Sponsor
Montefiore Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT04420585
Brief Title
Desmopressin for Bedwetting in Children With SCD
Official Title
Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Terminated
Why Stopped
We were not able to meet recruitment after 9 years of trying to enroll patients on different versions of the protocol and changing recruitment strategies.
Study Start Date
July 7, 2020 (Actual)
Primary Completion Date
June 12, 2023 (Actual)
Study Completion Date
June 12, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Montefiore Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No

5. Study Description

Brief Summary
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
Detailed Description
Night time bedwetting is a common complication of sickle cell disease, and affects up to 30 % of children . Desmopressin is an oral medication that increases water reabsorption in the kidneys. Studies have shown that it is effective in decreasing bedwetting episodes in children without sickle cell disease. Chronic sickling episodes causing damage to the kidneys could cause permanent damage and may make this treatment ineffective in sickle cell disease. This trial will inform pediatric sickle cell doctors if desmopressin is an appropriate treatment for bed wetting in the investigators patients. This is an edited continuation of study ID: 2014-3768.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Nocturnal Enuresis, Anemia, Sickle Cell

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment Group
Arm Type
Experimental
Arm Description
Desmopressin 0.2mg tablets, dose titrated to effect
Intervention Type
Drug
Intervention Name(s)
Desmopressin
Other Intervention Name(s)
DDAVP
Intervention Description
Two desmopressin 0.2 mg tablets at bedtime for 14 days and monitoring if <50 % improvement
Primary Outcome Measure Information:
Title
Change in Bedwetting episodes
Description
To prospectively assess if the use of desmopressin in patients with sickle cell disease and nocturnal enuresis will decrease the number of nighttime episodes of enuresis by 50% after initiating DDAVP at 0.4 mg nightly dose with dose escalation as clinically appropriate.
Time Frame
Baseline and 4 weeks
Secondary Outcome Measure Information:
Title
Impact of Bedwetting on day to day activities
Description
To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have an improved quality of life compared to their baseline. This will be measured using the PedsQL Measurement Model which measures health related quality of life in children with acute and chronic health conditions, like sickle cell. The scales focuses on areas such as activities, feelings, and school performance.
Time Frame
Baseline and 4 weeks
Title
Change in Nighttime awakenings
Description
To determine if the use of desmopressin in patients with nocturnal enuresis improves rates of nocturia, defined as episodes of nighttime awakening to void in children ≥5 years of age, compared to prior to initiating treatment with DDAVP. Patient's will track their nighttime awakenings using a study diary.
Time Frame
Baseline and 4 weeks
Title
Change in Daytime Fatigue
Description
To determine if patients with sickle cell disease and nocturnal enuresis receiving desmopressin will have less daytime fatigue compared to their baseline data. The PROMIS Pediatric Fatigue Short Form will be used to compare levels of fatigue from baseline to 4 weeks on the medication.
Time Frame
Baseline and 4 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with Hemoglobin SS, SC, SB0thal or SB+thal Patients with at least two episodes of primary nocturnal enuresis per week or four episodes over the two weeks prior to enrollment. Patients with secondary enuresis who have been evaluated and cleared by a pediatric urologist as not having other etiologies of enuresis (e.g. overactive detrusor activity, a genitourinary anatomic abnormality) Exclusion Criteria: Patients with developmental delay or neurologic dysfunction secondary to stroke. Patients with hypertension or underlying renal disease. Patients with genitourinary anatomic abnormalities. Any prior renal ultrasound showing normal genitourinary anatomy is sufficient to clear a patient for the study. Patients with daytime urinary incontinence Patients with glucosuria on urinalysis. Patients with secondary nocturnal enuresis who have not been evaluated by a pediatric urologist to rule out other etiologies of enuresis. Patients who are pregnant. Patients receiving another medicine for nocturnal enuresis (e.g. imipramine).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kerry Morrone, MD
Organizational Affiliation
Montefiore Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital at Montefiore
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
16753432
Citation
Neveus T, von Gontard A, Hoebeke P, Hjalmas K, Bauer S, Bower W, Jorgensen TM, Rittig S, Walle JV, Yeung CK, Djurhuus JC. The standardization of terminology of lower urinary tract function in children and adolescents: report from the Standardisation Committee of the International Children's Continence Society. J Urol. 2006 Jul;176(1):314-24. doi: 10.1016/S0022-5347(06)00305-3.
Results Reference
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PubMed Identifier
11718233
Citation
Barakat LP, Smith-Whitley K, Schulman S, Rosenberg D, Puri R, Ohene-Frempong K. Nocturnal enuresis in pediatric sickle cell disease. J Dev Behav Pediatr. 2001 Oct;22(5):300-5. doi: 10.1097/00004703-200110000-00004.
Results Reference
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PubMed Identifier
2334206
Citation
Readett DR, Morris JS, Serjeant GR. Nocturnal enuresis in sickle cell haemoglobinopathies. Arch Dis Child. 1990 Mar;65(3):290-3. doi: 10.1136/adc.65.3.290.
Results Reference
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PubMed Identifier
18384865
Citation
Field JJ, Austin PF, An P, Yan Y, DeBaun MR. Enuresis is a common and persistent problem among children and young adults with sickle cell anemia. Urology. 2008 Jul;72(1):81-4. doi: 10.1016/j.urology.2008.02.006. Epub 2008 Apr 2.
Results Reference
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PubMed Identifier
22999692
Citation
Naitoh Y, Kawauchi A, Soh J, Kamoi K, Miki T. Health related quality of life for monosymptomatic enuretic children and their mothers. J Urol. 2012 Nov;188(5):1910-4. doi: 10.1016/j.juro.2012.07.012. Epub 2012 Sep 19.
Results Reference
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PubMed Identifier
12137645
Citation
Glazener CM, Evans JH. Desmopressin for nocturnal enuresis in children. Cochrane Database Syst Rev. 2002;(3):CD002112. doi: 10.1002/14651858.CD002112.
Results Reference
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PubMed Identifier
21203778
Citation
Becker AM. Sickle cell nephropathy: challenging the conventional wisdom. Pediatr Nephrol. 2011 Dec;26(12):2099-109. doi: 10.1007/s00467-010-1736-2. Epub 2011 Jan 4.
Results Reference
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PubMed Identifier
5435231
Citation
Statius van Eps LW, Schouten H, Haar Romeny-Wachter CC, La Porte-Wijsman LW. The relation between age and renal concentrating capacity in sickle cell disease and hemoglobin C disease. Clin Chim Acta. 1970 Mar;27(3):501-11. doi: 10.1016/0009-8981(70)90305-0. No abstract available.
Results Reference
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PubMed Identifier
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Citation
Figueroa TE, Benaim E, Griggs ST, Hvizdala EV. Enuresis in sickle cell disease. J Urol. 1995 Jun;153(6):1987-9.
Results Reference
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PubMed Identifier
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Citation
Robson WL, Leung AK, Norgaard JP. The comparative safety of oral versus intranasal desmopressin for the treatment of children with nocturnal enuresis. J Urol. 2007 Jul;178(1):24-30. doi: 10.1016/j.juro.2007.03.015. Epub 2007 May 11.
Results Reference
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Desmopressin for Bedwetting in Children With SCD

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